https://www.haystacksci.com/home daily 1.0 2025-06-05 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/db41bc2e-36e5-4b4e-a74a-c6add7df4d37/unsplash-image-gKUC4TMhOiY.jpg Bridging Discovery and the clinic - Science commercialization https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/d0bba96e-713c-41c8-b72f-4bb4d0e97996/unsplash-image-2GEr4fLZt8A.jpg Bridging Discovery and the clinic - Ultra-rare diseases https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1749139088663-P07M4LCJAXI9Y5D1XU6M/unsplash-image-9Mq_Q-4gs-w.jpg Bridging Discovery and the clinic - The Needle Our Newsletter https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1749155424564-MPFSA0VT0JSUG31TP12A/unsplash-image-OKLqGsCT8qs.jpg Bridging Discovery and the clinic - The Mixer Our podcast https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/fc61b60f-49e3-4345-b9de-18b50bc3d8a0/unsplash-image-Skcr6X_aLjU.jpg Bridging Discovery and the clinic - Literature Our publications https://www.haystacksci.com/about daily 0.75 2025-01-17 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/83b94872-055d-4d69-8ca6-689f06a809f3/image-asset.jpg About Haystack Science https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/80175c51-1d89-4e87-b03a-e40a76daae1c/AM_Headshot2Cropped.jpg About Haystack Science - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.haystacksci.com/contact daily 0.75 2026-03-20 https://www.haystacksci.com/translational-masterclasses daily 0.75 2018-08-14 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1508946005412-VD2F6DE5X12G80AII1QF/jonathan-velasquez-384018.jpg from bench to bedside https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1508946064242-C8KNK7R8GO3J7TA1MKZM/mikael-kristenson-242070.jpg from bench to bedside https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1508946110387-9K6A0TBSX1A982UWLDRF/jonathan-simcoe-88013.jpg from bench to bedside https://www.haystacksci.com/editorial-masterclasses daily 0.75 2018-08-14 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1534101433713-2BL6R95CPJZCAE546Z0I/marten-bjork-620139-unsplash.jpg Demystifying the publishing process https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1534101450419-WTB92OIBW8HYMLUB26PF/antique-brand-business-163084.jpg Demystifying the publishing process https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1534101521354-IC7S63SQNXWDVBH2YEMG/neonbrand-618320-unsplash.jpg Demystifying the publishing process https://www.haystacksci.com/publishing-services daily 0.75 2018-08-24 https://www.haystacksci.com/science-commercialization daily 0.75 2026-03-20 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/32b6d36d-8456-4d92-aeb5-fb0aa5ace3f1/unsplash-image-5fNmWej4tAA.jpg Bridging Discovery and the market Technology-transfer offices https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/10d28c7a-3a8e-43b7-90cb-95a8d79fa565/unsplash-image-ToN8TFvS1XE.jpg Bridging Discovery and the market Research foundations https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/18f687c0-9cb3-427d-839f-8c009ed48f23/unsplash-image-mKYf6jV-rYo.jpg Bridging Discovery and the market Investors https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/7c9ff3f0-ceed-4544-8c78-09cd4f5aecd3/unsplash-image-PP5nO5gcLdA.jpg Bridging Discovery and the market - 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Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/907d6b56-69be-443e-ac54-3018bb128260/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/47fef395-2ad9-4040-a794-95c4d9106974/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/22f3edf4-05c2-4cba-8fd2-53b6b8d72983/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/1588631777110-V9CH3QRXNJDC4YON8LXU/Haystack+Thubmnail+Dark.png Literature - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.haystacksci.com/rras daily 0.75 2026-04-02 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/33af35f8-d2ab-48e8-8f12-3972c7883644/RRA+MEF2C+haploinsufficiency.jpg Ultra-rare diseases - Make it stand out MEF2C Haploinsufficiency Syndrome. This condition, caused by mutations in one copy of the transcription factor MEF2C, results in severe developmental delay, epilepsy and sleep disturbances, among other symptoms. Our research-readiness assessment shows that, although the disease mechanism is increasingly well understood, key gaps include the paucity of rescue data in preclinical models using any disease-modifying therapeutic modality and the absence of clinical-trial infrastructure. April 2, 2026 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/3e1d438f-d07e-49c7-b712-fbdad3f305c0/RRA+Shwachman-Diamond+Syndrome.jpg Ultra-rare diseases - Make it stand out Shwachman-Diamond Syndrome. This multi-system disorder is caused by mutations in the SBDS gene, a protein important for ribosomal biogenesis. The disease is characterized by bone-marrow failure, pancreatic insufficiency, skeletal abnormalities and other phenotypes. The main gaps that we identified pertain to the lack of in vivo data showing phenotypic rescue after therapeutic interventions. The emergence of new experimental models may help address this issue. March 30, 2026 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/3d4f5717-a7ca-4417-af9a-19d04cfecc2e/RRA+Timothy+Syndrome.jpg Ultra-rare diseases - Make it stand out Timothy Syndrome. This channelopathy is characterized by the presence of arrhythmias and neurodevelopmental phenotypes. Timothy Syndrome is caused by gain-of-function mutations in CACNA1C, which encodes the voltage-gated calcium channel Cav1.2. Our analysis shows that proof-of-concept data remain scarce, with ASOs being the preferred modality so far. Key gaps also remain in terms of clinical readiness and establishing the prevalence of the syndrome. March 26, 2026 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/128c4145-8881-46c7-9383-e8ac806a2bc1/RRA+PACS1+Syndrome.jpg Ultra-rare diseases - Make it stand out PACS1-related syndrome. Also known as Schuurs-Hoeijmakers Syndrome, this neurodevelopmental disorder is caused by mutations in a protein involved in trafficking through the Golgi network. The existence of strong genotype-phenotype correlations point to ASOs as the ideal therapeutic modality for this disease. However, important gaps remain on the clinical-development side. March 23, 2026 https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/8c76450a-238a-4c79-b953-1e060f8033f8/RRA+Gorlin+Syndrome.jpg Ultra-rare diseases Gorlin Syndrome. Condition caused by mutations in PTCH1 and characterized by the development of basal cell carcinomas and other tumors, as well as skeletal abnormalities. Our analysis shows very few gaps, which is not unexpected, considering the availability of therapies for this condition. But as the existing therapies are not curative, Gorlin Syndrome may be fertile ground for the development of advanced therapeutics. March 19, 2026. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/6d8aea91-a9c8-48a1-827b-86091742056a/ADSSL1+Myopathy.jpg Ultra-rare diseases ADSSL1 Myopathy. Mutations in the ADSSL1 gene, which encodes adenylosuccinate synthetase 1, affect purine metabolism and energy production in muscle cells. Our analysis shows that multiple gaps remain across the different domains that we evaluate, starting with a limited understanding of disease biology. March 16, 2026. https://images.squarespace-cdn.com/content/v1/58d2e59cbe65946d27801d46/524defd8-85e9-471b-b800-0ef0dc17566d/Alstrom+Syndrome.jpeg Ultra-rare diseases Alström Syndrome. Multi-system disorder caused by mutations in the ALMS1 gene. Our analysis indicates that important gaps remain in understanding disease mechanisms and in the identification of the ideal modality to develop a therapeutic. March 12, 2026.